This compilation allows readers to adjust their thinking to appreciate the full impact certain select technologies will make on the industry by 2026.

Six Approaches Making the Most Significant Impact in the Future

The testing of new convecdiff membranes shows this material to be well suited for lab- and commercial-scale bind-and-elute applications.

Purification of Antibodies Using Novel Convecdiff Membranes Part 1

Cautiously setting aside pandemic practices, pharma workers appear ready for flux, change, evolution, and expanding molecular diversity.

Crossing the Same River Multiple Times

Biologic drug development requires relevant bioassays to measure and help predict cellular response.

Complex Analytical Workflows Increasingly Require Robust Bioassays

Affinity chromatography resins must perform well under mild elution conditions yet withstand robust cleaning and sanitization protocols.

Choosing the Right Resins for Viral Vector Affinity Chromatography

Efforts to optimize the ideal blend of technology and best practices that are needed to protect and deliver time- and temperature-sensitive biologic therapies, vaccines, and cell and gene therapies continue to evolve.

Safeguarding Biologics— Blending Engineering Ingenuity and Managerial Excellence

Health crises, political tension, and budget concerns were major challenges.

Pandemic and Politics Shaped Pharma and FDA in 2022

Vetter announced on Jan. 18, 2023 that it had achieved gold status in the EcoVadis sustainability ranking, with 72 points out of a maximum of 100. This makes the company among the top 5% of all participating companies in the industry. The new ranking adds to the company’s commitment to implementing improvements in the rating criteria of Environment, Labor & Human Rights, Ethics, and Sustainable Procurement.

"We are very proud that our far-reaching initiatives in many areas around sustainable corporate governance have had a significant impact on the EcoVadis rating, and that we were, once again, able to demonstrate improvement over previous years. The positive external evaluation is a strong achievement and further motivates us to continue this great development in a transparent manner among our employees, customers, and partners, as well as the patients and society," said Peter Soelkner, managing director at Vetter.

"Simply talking about this topic is never enough in our opinion," added Thomas Otto, managing director at Vetter. "With our activities involving social, economic and ecological sustainability, we want to have a positive impact on the regions with our sites."

Articles

Vetter significantly increases its sustainability ranking by achieving gold status in EcoVadis ranking. 

Vetter Wins Gold in EcoVadis Ranking

Pfizer and Gero, a Singapore-based preclinical-stage physics-enabled biotechnology company, have entered into a research collaboration in which Pfizer will apply Gero’s machine learning technology platform to discover potential therapeutic targets for fibrotic diseases. The collaboration will use large-scale human-based data.

Under the agreement, Gero will receive an upfront payment. The company is also eligible to receive discovery milestone payments if the project progresses. The collaboration will leverage Pfizer's expertise and Gero’s technology platform to identify genes and pathways that are linked to fibrotic diseases. Pfizer may advance the potential therapeutic targets and would be responsible for any further preclinical and clinical development.

“Human data-driven drug discovery avoids the ‘preclinical trap’ and has the potential to identify clinically relevant targets against human (not rodent) diseases. However, genetics-based target identification against age-related diseases is hindered by the irreversible component of human aging,” said Peter Fedichev, CEO of Gero, in a Jan.6, 2023 company press release. “Our technology platform allows us to separate irreversible effects of aging from reversible disease phenotypes to potentially identify the most actionable therapeutic targets.”

“We are excited to work with Pfizer … to potentially identify targets against fibrotic diseases, which have a large unmet need. We believe that combining our platform technology with Pfizer’s extensive disease expertise has the potential to identify high-value targets in this disease area,” said Alex Kadet, chief business officer, Gero, in the release.

Pfizer and Gero have partnered to discover potential therapeutic targets for fibrotic diseases using machine learning technology.

Pfizer and Gero Form Collaboration to Discover Potential Targets for Fibrotic Diseases

AbbVie and US-based Anima Biotech (Anima) have formed a collaboration to discover and develop messenger RNA (mRNA) biology modulators for three targets across oncology and immunology. In the collaboration, Anima will use its mRNA Lightning technology platform to discover novel mRNA biology modulators against the collaboration targets. The company will provide AbbVie exclusive rights to license and further develop and commercialize these programs, according to a Jan. 10, 2023 company press release.

Under the agreement, Anima receives $42 million in an upfront payment and may be eligible to receive up to $540 million in option fees and R&D milestones in the aggregate across the three targets. The company will also have the potential to receive further commercial milestones and tiered royalties on net sales. AbbVie has an option to expand the collaboration with up to three additional targets under the same terms as the initial collaboration, which may increase the potential value of the collaboration.

"This collaboration will give AbbVie access to Anima's leading technology platform and deep expertise in mRNA biology and will help further strengthen AbbVie's [capabilities] in discovering and developing drugs to make a difference in patient[s’] lives," said Jonathon Sedgwick, vice-president and global head of discovery research, AbbVie, in the press release. "Modulating mRNA biology with small molecules is a new approach and has the potential to address 'undruggable' targets with implications across multiple therapy areas."

"Anima's differentiated approach in the field of small-molecule mRNA drugs combines phenotypic screening with AI [artificial intelligence]-driven elucidation of the mechanisms of action. The power of our mRNA Lightning platform is validated by our multiple partnerships and a growing internal pipeline," said Yochi Slonim, co-founder and chief executive officer, Anima, in the release.

The collaboration between AbbVie and Anima Biotech will combine Anima's mRNA Lightning technology platform with AbbVie's extensive expertise in oncology and immunology.

AbbVie and Anima Biotech Partner on Discovery and Development of mRNA Biology Modulators

FDA published a guidance document on Jan. 16, 2023 intended to assist applicants in developing the Dosage and Administration section of labeling as described in 21 

201.57, a regulation governing the content and format of the section of human prescription drug and biological product labeling. The applicants should follow the recommendations in the new guidance when developing the Dosage and Administration section for a new drug submitted to FDA under a new drug application under section 505(b) of the Food, Drug, and Cosmetic Act or a biologics license application under section 351(a) of the Public Health Service Act.

The guidance provides examples of required and recommended information in the Dosage and Administration section.

FDA Publishes Guidance on Labeling Drugs and Biological Products 

FDA signed an agreement on Mutual Recognition between the Swiss Confederation and the United States Relating to pharmaceutical good manufacturing practice (GMP). The agreement will be able to let FDA and the Swiss Agency for Therapeutic Products (Swissmedic) utilize each other’s GMP inspections of manufacturing facilities, avoiding the need for duplicate inspections.

The Food and Drug Administration Safety and Innovation Act, enacted 2012, permits FDA to enter into agreements to recognize drug inspections conducted by foreign regulatory authorities determined to be capable of conducting inspections that meet US requirements. Mutual Recognition Agreements (MRAs) are already in place by FDA with the European Union and the United Kingdom. The MRA with Swissmedic includes veterinary drugs, in addition to covering GMP inspections of facilities making human drugs.

Further, FDA’s Office of Global Policy and Strategy has been negotiating the US-Switzerland MRA for several years. Before the MRA is in control, FDA must determine whether Swissmedic can conduct inspections that meet US requirements. Similarly, Swissmedic must decide with respect to FDA meeting Swiss requirements.

“In today’s global pharmaceutical market, MRAs offer a way for the FDA to work more efficiently and maximize its resources,” said Andi Lipstein Fristedt, FDA deputy commissioner for Policy, Legislation, and International Affairs, who signed the agreement on behalf of the FDA, in a company press release. “Once the MRA enters into force, the FDA will be able to rely on the factual findings of Swissmedic experts in many cases, thus avoiding duplicate inspections and allowing the FDA to expand its inspectional reach.”

The agreement will be able to let FDA and the Swiss Agency for Therapeutic Products (Swissmedic) utilize each other’s GMP inspections of manufacturing facilities, avoiding the need for duplicate inspections.

FDA Signs Mutual Recognition Agreement with Swiss Confederation 

At the European Medicines Agency’s (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) meeting on Jan. 12, 2023, the members reviewed the measures to minimize the risk of serious side effects associated with Janus kinase (JAK) inhibitors used to treat several chronic inflammatory disorders. According to the PRAC, the use of lower dose of Olumiant (baricitinib) for patients at higher risk of blood clots, cardiovascular conditions, and cancer is in line with the dosing recommendations for other JAK inhibitors, such as Rinvoq (updacitinib), Cibinqo (abrocitinib), and Jyseleca (filgotinib). 

As for new safety information for healthcare professionals, the PRAC focused on direct healthcare professional communication (DHPC) containing important information on Zolgensma (onasemnogene abeparvovec).

Zolgensma, a gene therapy medicine for the treatment of spinal muscular atrophy, has recently reported fatal cases of acute liver failure in patients treated with it. The DHPC lets healthcare professionals know of the fatal cases of liver failure and the updated recommendations for monitoring liver function, assessing suspected liver injury after infusion, and further advice regarding narrowing the corticosteroid treatment.

The updated DHPC for Zolgensma will be sent to EMA’s committee for advanced therapies and to EMA’s human medicines committee. After being adopted, the DHPC will be disseminated to healthcare professionals by the marketing authorization holders and published on the direct healthcare professional communications’ page and in national registers in EU Member States.

The members reviewed the measures to minimize the risk of serious side effects associated with Janus kinase (JAK) inhibitors used to treat several chronic inflammatory disorders. 

Pharmacovigilance Risk Assessment Committee Meeting Highlights

Veeva Systems announced on Jan. 13, 2023 that Adare Pharma Solutions, a contract development and manufacturing (CDMO) with expertise in oral dosage forms, chose Veeva Vault Quality Suite to harmonize quality systems across the organization. This choice can help Adare streamline quality processes, work more effectively with partners and suppliers, and deliver greater visibility into operations to customers.

“As Adare expands its business, standardizing on the technology many of our customers use is a top priority to streamline collaboration,” said Audrey Butler, vice president, global head of quality at Adare Pharma Solutions, in a press release. “Veeva Vault Quality Suite will bring together our global teams on one platform for seamless processes and real-time data access that can help us improve how we work with clients and deliver differentiated services.”

The Vault Quality Suite will help Adare simplify its quality operations across its sites in the United States, France, and Italy. Further, Adare will use Veeva Vault QMS to manage all quality processes, Veeva Vault QualityDocs to control documents and ensure quality, and Veeva Vault Training to drive employee qualifications. This new collaboration helps Adare to manage content, data, and training in a single end-to-end quality system.

“We’re proud to partner with Adare Pharma Solutions to help support their goals of improving services for clients and delivering transformational medicines for patients,” said Ashley Wentworth, senior director, Veeva Vault Quality strategy, in a press release. “With the Veeva Vault Quality Suite, Adare will have a scalable cloud platform that can keep up with evolving industry and customer requirements.”

The Vault Quality Suite will help Adare simplify its quality operations across its sites in the United States, France, and Italy.

Adare Pharma Solutions Coordinating Quality with Veeva Vault Quality Suite

Boehringer Ingelheim and 3T Biosciences announced on Jan. 13, 2023 that they have entered into a strategic collaboration and licensing agreement to discover and develop next-generation cancer therapies to address high unmet patient needs.

The partnership is meant to unite 3T Biosciences’ best-in-class 3T-TRACE (T-Cell Receptor Antigen and Cross-Reactivity Engine) discovery platform with Boehringer Ingelheim’s two-pronged research strategy combining cancer cell-directed and immune cell-targeting compounds, further strengthening the pipeline.

The platform from 3T platform aims to address this challenge by identifying novel shared T-cell receptor (TCR) targets of productive immune responses and comprehensively screening TCRs and TCR mimetics for specificity and off-target cross-reactivities. It is intended to identify the most prevalent and immunogenic targets in solid tumors by uniquely combining high-diversity target libraries with active machine learning. This could lead to tumor-specific, safer therapies that can be delivered at high doses.

“At Boehringer Ingelheim, we are committed to transforming patients’ lives. Through our new partnership with 3T Biosciences, we aim at accelerating and expanding our pipeline of first-in-class T-cell based therapies for patients affected by cancer,” said Lamine Mbow, PhD, global head of Cancer Immunology + Immune Modulation, Boehringer Ingelheim, in a press release.

Stefan J. Scherer, MD, PhD, president and CEO of 3T Biosciences, added that 3T’s 3T-TRACE discovery platform has the potential to transform the treatment of cancers and beyond. “By using data from patients for patients we aim to discover the best immunogenic targets for multiple tumor indications and across patient populations,” he said in a press release.

The agreement notes that Boehringer Ingelheim will provide patient-derived TCR data to fuel 3T’s target discovery efforts to identify antigens using its 3T TRACE discovery platform. Additionally, 3T will receive an upfront payment and research and development support, while still being eligible for discovery, preclinical, clinical, regulatory, and commercial milestones for a total of $268 million and royalties on future Boehringer Ingelheim product sales.

It is intended to identify the most prevalent and immunogenic targets in solid tumors by uniquely combining high-diversity target libraries with active machine learning.

Boehringer Ingelheim, 3T Biosciences Join Forces to Develop Next-Generation Cancer Immunotherapies

The new oral suspending base from PCCA, called SuspendIt Anhydrous, can be used on APIs that are unstable in water or are incompatible with existing aqueous vehicles, according to a company press release.

“An anhydrous base offers many benefits to compounding pharmacies,” said Gus Bassani, PCCA chief scientific officer, in the press release. “There’s no water to support the growth of micro-organisms or cause hydrolytic degradation of an API and it opens the potential for formulations to be assigned longer beyond-use dates (BUDs). But what makes our new base really unique is its self-emulsifying properties. This self-emulsifying system improves the miscibility and dispersibility of formulations in aqueous environments and may improve API solubility as well.”

Many anhydrous bases are oil-based; however, many patients do not like the taste of oil-based vehicles or the oily residue left in their mouths and throats. These highlighted issues along with other problems have led to problems with medication compliance.

“We developed a unique anhydrous base that, in addition to the self-emulsifying properties, has an improved viscosity, which reduces when shaken and thickens when standing. This thixotropic effect allows for rapid redispersion of APIs with agitation and minimizes sedimentation,” said Daniel Banov, PCCA director of Research and Development, in a press release. “We added a natural sweetener, as well as the ability to mix the compounded medicine with juice or water at the moment of administration, for a better taste if desired. The technology is so unique that we have a patent pending.”

PCCA has also created formulas for the most prescribed APIs and plans to continue formula development and API testing.

This self-emulsifying system improves the miscibility and dispersibility of formulations in aqueous environments and may improve API solubility.

SuspendIt Anhydrous Introduced as New Compounding Base

Spectrum Laboratory Products announced on Jan. 9, 2023 that the company is voluntarily recalling three lots of epinephrine (L-Adrenaline) USP, a bulk API used to manufacture or compound prescription products to the user level.

The L-Adrenaline USP bulk API powder is used in manufacturing and compounding of finished-dose epinephrine prescription products that can be used to treat a variety of medical conditions including anaphylaxis and other severe immediate hypersensitivity reactions, asthma, bronchospasm, airway edema, nasal congestion, dilation during intraocular surgery, vasoconstrictor with local anesthetics, hypotension or shock, heart failure, bradycardia or atrioventricular block, and sudden cardiac arrest.

Epinephrine is a critical medication used during life-threatening conditions that can affect any age and any person. The use of a finished-dose product manufactured or compounded with the recalled product could result in less-effective product and incomplete treatment of life-threatening conditions, such as low blood pressure, heart failure, anaphylaxis, irregular heartbeat, and heart attack. Further, treatment with a less-effective product or underdosing of epinephrine could result in death.

The main customer complaints have found that the product is discolored. The doses that are included in the impacted Epinephrine USP product can be identified by Spectrum catalog number EP130.

There have not been any reports of adverse events related to this recall, according to Spectrum Laboratory Products.

Epinephrine is a critical medication used during life-threatening conditions that can affect any age and any person. 

Spectrum Laboratory Products Issues Recall of Epinephrine USP Bulk API Due to Discoloration

A FDA guidance was published on Jan. 4 2023 that provides recommendations for the format and content of a risk evaluation and mitigation strategy (REMS) document for a prescription drug product and a biological drug product. A REMS document is a part of a REMS required by FDA and establishes the goals and requirements of the REMS.

This guidance specifically provides recommendations to applicants on drafting proposed REMS documents and converting an already-approved REMS document to a new, standardized format that is clearer and more informative while supporting the submission of a REMS document in Structured Product Labeling format.

An overview of the types of information that should be included in a REMS document is provided in the new document, whereas additional and more detailed information is provided in a separate guide, titled 

REMS Document Technical Conformance Guide

, which will be updated periodically on FDA’s website.

The guide can be used for drafting a REMS document for single product and shared system REMS and includes an outline for drafting a Bifurcated4 REMS document.

A REMS document is a part of a REMS required by FDA and establishes the goals and requirements of the REMS.

Updated REMS Document for Prescription, Biological Drug Product

AstraZeneca announced a definitive agreement to acquire CinCor Pharma, a clinical-stage biopharmaceutical company focused on developing novel treatments for hypertension and chronic kidney disease, on Jan. 9, 2023. The deal, worth up to $1.8 billion, grants AstraZeneca global rights to baxdrostat (CIN-107), a novel aldosterone synthase inhibitor (ASI) currently in development as a treatment to lower blood pressure in treatment-resistant hypertension. The deal is expected to close in the first quarter of 2023.

According to a company press release, CIN-107 is highly selective for aldosterone synthase and spares the cortisol pathway in humans. AstraZeneca will also acquire the rights to farxiga (dapagliflozin), another treatment designed to prevent and delay cardiorenal disease.

“Acquiring CinCor supports our commitment to cardiorenal disease and further strengthens our pipeline with [CIN-107],” said Mene Pangalos, executive vice president, BioPharmaceuticals R&D, AstraZeneca, in the press release. “Excess levels of aldosterone are associated with hypertension and several cardiorenal diseases, including chronic kidney disease and coronary artery disease and being able to effectively reduce this would offer a much-needed treatment option for these patients.”

“We are excited about the proposed acquisition of CinCor Pharma by AstraZeneca as we believe it offers the prospect of accelerating the development timeline and expanding the breadth of benefits patients with cardiorenal diseases might obtain from baxdrostat, if approved,” said Marc de Garidel, CEO, CinCor, in the press release. “CinCor is committed to ensuring a smooth transition of the development responsibilities to AstraZeneca once the acquisition is consummated.”

The definitive agreement will give AstraZeneca global rights to CinCor’s portfolio of cardiorenal disease treatments.